- SUNFISH Part 2 study population includes broad range of ages and disease severities, representing a real-world spectrum of people living with Type 2 or 3 SMA
- Evrysdi is the first and only at home SMA treatment approved by the FDA, and has proven efficacy across adults, children and infants 2 months and older
- More than 2,500 patients now treated with Evrysdi in clinical trial, compassionate use and real-world settings
“These results build on the one-year findings from the SUNFISH trial and importantly show the durability of improvement or stabilization of motor function through two years of treatment,” said SUNFISH principal investigator
Patients in SUNFISH Part 2 ranged in age from 2-25 and were treated with Evrysdi (n=120) or placebo and Evrysdi (n=60; patients in the placebo arm received placebo for 12 months followed by Evrysdi treatment for 12 months). The study evaluated a number of exploratory 24-month endpoints, which provide important insights into motor function and its impact on daily life. Findings demonstrated that Evrysdi:
- Maintained motor function improvements between months 12 and 24 as measured by Motor Function Measure (MFM-32)*.
- Increased motor function as measured by Revised Upper Limb Module (RULM)** and the Hammersmith Functional Motor Scale-Expanded (HFMSE)*** between months 12 and 24.
- Stabilized motor function for patients who began treatment with Evrysdi after 12 months of placebo as measured by MFM-32, RULM and HFMSE.
- Increased total score change from baseline, as measured by the caregiver-reported SMAIS**** upper limb module, and the patient-reported SMAIS score stabilized between months 12 and 24.
“These encouraging results confirm that the efficacy and safety of Evrysdi in people with Type 2 and Type 3 SMA can be sustained over time,” said
Decreases in serious adverse events, high-grade adverse events and treatment-related adverse events were observed in the second year versus the first year in both treatment arms. The most common adverse events observed in the Evrysdi arm and the placebo and Evrysdi arm from 12-24 months were upper respiratory tract infection (15.8% and 10%, respectively), nasopharyngitis (21.7% and 16.7%, respectively), pyrexia (13.3% and 10%, respectively), headache (10% and 16.7%, respectively), diarrhea (7.5% and 10%, respectively), vomiting (11.7% and 13.3%, respectively) and cough (10% and 8.3%, respectively). The most common serious adverse events were pneumonia (6.7% and 0%, respectively) and influenza (0.8% and 0%, respectively).
About Evrysdi™ (risdiplam)
Evrysdi is a survival of motor neuron 2 (SMN2) splicing modifier designed to treat SMA by increasing and sustaining production of the survival of motor neuron (SMN) protein. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement. Evrysdi is administered daily at home in liquid form by mouth or by feeding tube.
The
Evrysdi is currently being evaluated in four multicenter trials in people with SMA:
- FIREFISH (NCT02913482) – an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose-escalation study in 21 infants with the primary objective of assessing the safety profile of Evrysdi in infants and determining the dose for Part 2. Part 2 is a pivotal, single-arm study of Evrysdi in 41 infants with Type 1 SMA treated for 2 years, followed by an open-label extension. Enrollment for Part 2 was completed in
November 2018 . The primary objective of Part 2 was to assess efficacy as measured by the proportion of infants sitting without support after 12 months of treatment, as assessed in the Gross Motor Scale of the Bayley Scales of Infant andToddler Development – Third Edition (BSID-III) (defined as sitting without support for 5 seconds). The study met its primary endpoint. - SUNFISH (NCT02908685) – SUNFISH is a two-part, double-blind, placebo controlled pivotal study in people aged 2-25 years with Types 2 or 3 SMA. Part 1 (n=51) determined the dose for the confirmatory Part 2. Part 2 (n=180) evaluated motor function using total score of Motor Function Measure 32 (MFM-32) at 12 months. MFM-32 is a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA. The study met its primary endpoint.
- JEWELFISH (NCT03032172) – an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi. The study has completed recruitment (n=174).
- RAINBOWFISH (NCT03779334) – an open-label, single-arm, multicenter study, investigating the efficacy, safety, pharmacokinetics and pharmacodynamics of Evrysdi in babies (~n=25), from birth to six weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. The study is currently recruiting.
About SMA
SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 babies and is the leading genetic cause of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly, leading to muscle weakness over time. Depending on the type of SMA, an individual’s physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.
About
Neuroscience is a major focus of research and development at
About
Founded in 1896,
The
All trademarks used or mentioned in this release are protected by law.
* MFM is a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA. It assesses 32 different motor functions from standing and walking through to use of hands and fingers.
** RULM is a scale designed to assess upper limb movement in people with SMA. It can capture progressive muscle weakness across the spectrum of the disease, reflective of the SUNFISH Part 2 study population.
*** HFMSE is intended to be used in assessing the functional motor abilities of people with SMA who are able to sit and walk.
**** SMAIS was developed to measure self-reported and caregiver-reported independence.
Roche Group Media Relations
Phone: +41 61 688 8888 / e-mail: media.relations@roche.com
Dr. Phone: +41 61 687 05 17 | Phone: +41 61 688 44 86 |
Dr. Phone: +41 61 688 31 10 | Phone: +41 61 682 28 31 |
Nina Mählitz Phone: +41 79 327 54 74 | Phone: +41 61 687 43 05 |
Dr. Phone: +41 61 687 89 67 |
Roche Investor Relations | |
Dr. Phone: +41 61 68-78503 e-mail: karl.mahler@roche.com | Phone: +41 61 68-83894 e-mail: jon_kaspar.bayard@roche.com |
Dr. Phone: +41 61 68-88027 e-mail: sabine.borngraeber@roche.com | Dr. Phone: +41 61 68-75284 e-mail: bruno.eschli@roche.com |
Dr. Birgit Masjost Phone: +41 61 68-84814 e-mail: birgit.masjost@roche.com | Dr. Phone: +41 61 68-72942 e-mail: gerard.tobin@roche.com |
Investor Relations | |
Phone: +1 650 225 3217 e-mail: kalm.loren@gene.com | Dr. Phone: +1 650 467 8737 e-mail: tuomi.lisa@gene.com |
Attachment
- 130321_MR_MDA Sunfish for Stage 3_EN
© OMX, source